Clearer, CRISPR vision

Although the eye is a prime organ for gene editing due to its easy accessibility and shielding from the immune system, many genetic disorders are so heterogeneous that designing agents to correct each gene is commercially unfeasible. A study from NIH’s National Eye Institute (NEI) exploits a new target in the disease, NRL, and uses CRISPR to create a therapeutic that could be generalizable to retinitis patients who harbor a variety of mutations.

The findings will likely be of interest to gene editing companies, at least two of whom - Editas Medicine Inc. (NASDAQ:EDIT) and Intellia Therapeutics Inc. (NASDAQ:NTLA) - have programs in ophthalmology. This week, Editas announced an R&D collaboration with Allergan plc (NYSE:AGN) to develop CRISPR therapeutics for ophthalmic disorders. Editas received $90 million to develop five programs, including its lead program for Leber Congenital Amaurosis (LCA) type 10, in exchange for an option to develop and commercialize each of the programs. ...